There is a significant gap between the large number (~3500) of rare diseases for which we know the responsible gene and the small number (~200) for which there is an available therapy; an asymmetry that, with the current pace of gene discovery, will only increase over time. Given that neither academia nor industry alone possesses the skill sets or resources to develop treatments for rare diseases, CARE for RARE is pleased to collaborate with the multiple pharmaceutical companies to maximize opportunities for treatment discovery and clinical translation.